← Back to Search

Other

AMX0035 for Lou Gehrig's Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by Amylyx Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up between day 1 and day 40
Awards & highlights

Study Summary

This trial will study the effects of a new drug for ALS in adults.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~between day 1 and day 40
This trial's timeline: 3 weeks for screening, Varies for treatment, and between day 1 and day 40 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Blood concentration of PB and taurursodiol
Systemic exposure to PB and taurursodiol
Secondary outcome measures
Effect of a fixed dose combination of sodium phenyl butyrate (PB) and taurursodiol on pharmacodynamic activity
Effect of demographic characteristics on blood concentration of PB and taurursodiol
Effect of demographic characteristics on systemic exposure of PB and taurursodiol

Side effects data

From 2019 Phase 2 trial • 137 Patients • NCT03127514
28%
Fall
21%
Diarrhoea
20%
Muscular weakness
18%
Nausea
15%
Headache
13%
Constipation
11%
Salivary hypersecretion
11%
Viral Upper Respiratory Tract Infection
10%
Dizziness
10%
Dyspnoea
8%
Abdominal pain
8%
Decreased appetite
8%
Fatigue
7%
Laceration
6%
Back pain
6%
Abdominal discomfort
6%
Muscle spasms
6%
Arthralgia
6%
Musculoskeletal pain
6%
Contusion
6%
Urinary Tract Infection
6%
Cough
6%
Weight decreased
6%
Asthenia
6%
Rash
4%
Aspartate aminotransferase increased
4%
Musculoskeletal chest pain
4%
Abdominal pain upper
4%
Upper Respiratory Tract Infection
4%
Alanine aminotransferase increased
3%
Dysphagia
3%
Dry mouth
3%
Insomnia
2%
Respiratory failure
2%
Neck pain
2%
Oedema peripheral
1%
Pneumoperitoneum
1%
Skull fracture
1%
Bacteraemia
1%
Cellulitis
1%
Pneumonia
1%
Diverticulitis
1%
Nephrolithiasis
1%
Respiratory arrest
1%
Vision blurred
1%
Subdural haematoma
1%
Stoma site haemorrhage
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
AMX0035

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment with AMX0035Experimental Treatment1 Intervention
Two sequential study period. In Period 1, subject receive AMX0035 daily for approximately 14 days. In Period 2, subjects receive AMX0035 twice a day, morning and evening, for up to 25 days.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AMX0035
2018
Completed Phase 3
~360

Find a Location

Who is running the clinical trial?

Amylyx Pharmaceuticals Inc.Lead Sponsor
9 Previous Clinical Trials
2,167 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
NeuropsychopharmacologyJournal
Phenylbutyrate Ameliorates Cognitive Deficit and Reduces Tau Pathology in an Alzheimer's Disease Mouse Model
Ricobaraza, Ana, Mar Cuadrado-Tejedor, Alberto Pérez-Mediavilla, Diana Frechilla, Joaquin Del Río, and Ana García-Osta. 2009. “Phenylbutyrate Ameliorates Cognitive Deficit and Reduces Tau Pathology in an Alzheimer's Disease Mouse Model”. Neuropsychopharmacology. Springer Science and Business Media LLC. doi:10.1038/npp.2008.229.
Journal of Biological ChemistryJournal
Phenylbutyrate Up-regulates the DJ-1 Protein and Protects Neurons in Cell Culture and in Animal Models of Parkinson Disease
Zhou, Wenbo, Kathryn Bercury, Jessica Cummiskey, Nancy Luong, Jacob Lebin, and Curt R. Freed. 2011. “Phenylbutyrate Up-regulates the DJ-1 Protein and Protects Neurons in Cell Culture and in Animal Models of Parkinson Disease”. Journal of Biological Chemistry. Elsevier BV. doi:10.1074/jbc.m110.211029.
Neurobiology of DiseaseJournal
Tauroursodeoxycholic Acid (TUDCA) Supplementation Prevents Cognitive Impairment and Amyloid Deposition in APP/PS1 Mice
Lo, Adrian C., Zsuzsanna Callaerts-Vegh, Ana F. Nunes, Cecília M.P. Rodrigues, and Rudi D'Hooge. 2013. “Tauroursodeoxycholic Acid (TUDCA) Supplementation Prevents Cognitive Impairment and Amyloid Deposition in APP/PS1 Mice”. Neurobiology of Disease. Elsevier BV. doi:10.1016/j.nbd.2012.09.003.
Neurobiology of AgingJournal
Amyloid-β Pathology Is Attenuated by Tauroursodeoxycholic Acid Treatment in APP/PS1 Mice After Disease Onset
Dionísio, Pedro A., Joana D. Amaral, Maria F. Ribeiro, Adrian C. Lo, Rudi D'Hooge, and Cecília M.P. Rodrigues. 2015. “Amyloid-β Pathology Is Attenuated by Tauroursodeoxycholic Acid Treatment in APP/PS1 Mice After Disease Onset”. Neurobiology of Aging. Elsevier BV. doi:10.1016/j.neurobiolaging.2014.08.034.
~4 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top